Overview

Iron-Chelating Therapy and Friedreich Ataxia

Status:
Completed
Trial end date:
2008-03-01
Target enrollment:
Participant gender:
Summary
Friedreich ataxia, an autosomal recessive condition, ascribed to frataxin gene expansion, has been shown to result from an iron- induced injury to the mitochondrial respiratory chain. Buffering free radicals with short-chain quinones (Idebenone) protects the patients against cardiomyopathy but not CNS involvement. Removing CNS iron should limit the impact of the neurological symptoms of the disease.
Phase:
Phase 1/Phase 2
Details
Lead Sponsor:
Assistance Publique - Hôpitaux de Paris
Treatments:
Iron
Iron Chelating Agents